Sentences with phrase «defective gene in the cells»
Our lead candidate, LYS - GM101, has been designed to replace this defective gene in the cells of GM1 patients, in order to allow for production of the functional lysosomal acid beta - galactosidase (bgal) enzyme.
http://www.lysogene.com/ Not exact matches
Where is the clear line in a progression from (1) using animal insulin to treat diabetes, to (2) using gene remodeling techniques to grow insulin in a host bacterium that will reproduce rapidly and from which a plentiful supply of insulin can be harvested, to (3) genetic surgery to replace the defective gene in a person diagnosed as diabetic, to (4) genetic surgery immediately after fertilization in order to replace the defective gene and alter the germ cells which would otherwise have transmitted the disease to one's offspring?
They destroyed the T cells in 12 mice, five of which received marrow cells from normal mice while seven received marrow from mice with a defective Fas - ligand gene.
«Techniques to correct defective genes in «non-reproductive» cells are already at various stages of clinical development and promise to be a powerful approach for many human diseases which don't yet have an effective treatment.
«Non-small cell lung cancers can be sorted in clusters by endocytic changes: Sorting cancer by defective endocytosis genes.»
When it's done inside the body, doctors may inject the virus carrying the gene in question directly into the part of the body that has defective cells.
Lombardo A, Genovese P, Beausejour CM, Colleoni S, Lee YL, Kim KA, Ando D, Urnov FD, Galli C, Gregory PD, Holmes MC, Naldini L. Gene editing in human stem cells using zinc finger nucleases and integrase - defective lentiviral vector delivery.
Other genetic diseases include Tay - Sachs disease (damage to the gene for the enzyme hexosaminidase A leads to an accumulation of a chemical in the brain that destroys it), sickle cell anemia (improper coding of the gene that produces hemoglobin), hemophilia (lack of a gene for a blood - clotting factor) and muscular dystrophy (caused by a defective gene on the X chromosome).
To study the effects of LRO - related gene mutations, Dr. Huizing is performing fluorescent protein expression studies using patients» cells in order to examine defective intracellular trafficking.
A mutant cell line defective in response to double - stranded RNA and in regulating basal expression of interferon - stimulated genes
It was established later (Johnson et al, 1980) that the form of the disease found in Bedlingtons terriers was apparently caused by the presence of an autosomal recessive gene in affected dogs - it is a genetic disorder and in order for the condition to develop in a dog / bitch a copy of this defective gene must have been passed to the affected animal by both parents, i.e. the affected dog must have two «copies» of the defective gene in its body cells.